The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. RGX-314, RGX-202, RGX-121, RGX-111, RGX-181, RGX-381. It is a recombinant adeno-associated virus serotype 9 (AAV9) capsid containing a shortened version of the human dystrophin gene (mini-dystrophin) controlled by a human muscle specific promotor. Arising in one of every 3,500 to 5,000 male infants worldwide, DMD is a rare neuromuscular disease caused by mutations in the gene encoding for the protein dystrophin. Additional design elements, such as codon optimization and CpG content reduction, have the potential to enhance gene expression, increase translational efficiency, and reduce immunogenicity. Importantly, there were no serious adverse events (only mild to moderate events). There are currently three companies with competitive trials in the US: Solid Biosciences, Sarepta Therapeutics, and Pfizer (who bought the DMD platform in 2016 Could we use histamine? Extensive pre-clinical evidence also formed part of the BLA. Founded in 1995, Sangamo Therapeutics is a biotech company based in Richmond, California that focuses on developing gene therapies for rare genetic diseases and cancer. Germline gene therapy, on the other hand, involves modifying genes in reproductive cells, such as eggs or sperm. Waiting in the wings is Pfizer, whose DMD hopeful PF-06939926encountereda roadblock late last year after a treated patient died. Cellectis has more than two decades of experience in gene editing. In November 2021, RGX-202 was designated as an orphan drug by the FDA for Duchenne Muscular Dystrophy treatment. This explains why it largely affects boys as they dont have a backup copy of the gene (they only have one X chromosome). Moreover, a thorough clinical assessment, a complete patient history, and a number of specialist diagnostics, including molecular genetic tests, are used for Duchenne Muscular Dystrophy diagnosis. Thats why the first DMD gene therapy trial in the US, which began in 2006, involved injecting the gene therapy directly into the biceps of the children who participated. They can help slow down disease progression, but tackling inflammation only addresses one downstream effect.. Regardless, Pfizer will need to examine the situation and acquire the data necessary to continue the Phase Ib trial and make changes to future trials, such as omitting certain mutation types. The company aims to develop therapies for neurological disorders and other diseases. Their gene therapy products are based on ex-vivo gene therapy, which involves modifying a patient's own cells outside the body and then reintroducing them. The first U.S. human gene therapy trial directed at Duchenne muscular dystrophy (DMD) was launched yesterday at Columbus (Ohio) The gene therapy is currently being evaluated in a late-stage clinical development trial for the Duchenne Muscular Dystrophy treatment. Adeno-associated viruses (AAVs) are commonly used because they dont naturally cause disease or many immune system side effects in humans. exa-cel, CTX110, CTX112, CTX130, CTX131, anti-CD83 autologous CAR-T, VCTX210, VCTX211, VCTX212, CTX310. He has extensive experience in the Bio-pharmaceutical field, with positions at Pfizer, as Medical Director of Rare Diseases and, more recently Moderna, where he was responsible for taking the first mRNA therapeutics for rare diseases into the clinic. Gene therapy; Cell therapy; Drug therapy; Mutation specific approaches; TREAT-NMD Services Limited is a wholly owned subsidiary of TREAT-NMD Alliance Limited, a registered charity in England & The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. It is also developing therapies for blood stem cell, immuno-oncology and regenerative medicine. The company was spun-out from University College London in 2014. Focuses on allogeneic placental-derived cells. WebThere are many challenges facing the management of DMD patients in the United Arab Emirates and Kuwait and most likely other countries within the Middle East. This may lead to dangerous side effects. Data are expected to start rolling in late next year. Recently Serepta released the topline results from Part 2 of Study SRP-9001-102 (Study 102), an ongoing, double-blind, randomized, placebo-controlled clinical study to assess the safety, efficacy, and tolerability of a single dose of SRP-9001 (delandistrogene moxeparvovec) in 41 patients with Duchenne Muscular Dystrophy, 21 of whom were in the placebo crossover cohort. Mondays BLA acceptance positions Roche and Sarepta as the leaders of a tight race to bring a gene therapy for DMD over the regulatory finish line. The FDA hasacceptedRocheandSareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an investigational gene therapy for Duchenne muscular dystrophy (DMD). Even if both gene therapies reach the market, PF-06939926 is likely to face a delay due to the recent death in its Phase Ib trial. The FDA has accepted Roche and Sareptas Biologic License Application for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec), an It is very likely that one or both of these gene therapies could be approved., This opens up the door for combination therapies, such as gene therapies to stabilize the muscle and small molecule drugs to deal with downstream events like fibrosis and inflammation, Hesterlee concluded. Cell and gene therapy companies also aim to cure common forms of blindness and restore sight in patients with inherited retinal diseases. The Food and Drug Administration approved the therapies after studying a few dozen boys. What about a tourniquet and pressure? Feb 18, 2022 | Reading Time: 8 minutes. Patients with this form of the muscle-wasting disease don't make enough dystrophin, a protein Which Pipeline Therapy Has The Potential To Revolutionize The Dystrophic Epidermolysis Bullosa Treatment Market? At the American Society of Gene and Cell Therapy Meeting, the companies theorized that the adverse events were most likely driven by the body's immune responses to the protein expressed by their gene therapeutic. https://www.pharmalive.com/wp-content/uploads/2021/08/Mega-3-Billion-Deal-Shapes-Up-for-Roche-to-Target-AD-and-Parkinsons-BioSpace-8-24-21.jpeg, https://www.pharmalive.com/wp-content/uploads/2020/01/Pharmalive_4c-300x37.png, FDA accepts BLA for Roche-Sarepta's DMD gene therapy, Copyright - PharmaLive and Outcomes LLC |, Axsome headed to FDA after Phase III Alzheimers agitation win, Social Determinants of Health (SDOH): Three Trends to Watch in 2023, U.S. Centers for Disease Control and Prevention (CDC). WebGene Therapy: Gene therapy for DMD uses selected portions of the dystrophin gene to create a smaller, potentially functional version. Because of its ability to target muscle tissue, the AAV9 capsid was chosen as the delivery mechanism and is administered intravenously. Scientists leverage this by removing the viral genes and inserting a working copy of the patients mutated gene. Web2 Department of Gene Therapy, Saad Pharmaceuticals, Tornime 7-26, Tallinn, 10145, Estonia. Pfizer Inc. Website: www.pfizer.com. The company is working with the Cas9 and Cas12a CRISPR nucleases. GlobalDatas report assesses how GALGT2 (Nationwide Childrens)s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. The earlier you treat, the better, but its hard to measure benefit if the children are not yet manifesting a lot of symptoms, so you want to test the children at a stage when theyre progressing, said Hesterlee. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). An IND application is expected by the end of 2021. Were still learning from human studies, it just shows that not every model will be predictive of the human clinical finding, he said. All rights reserved. The first signs of DMD appear as the young boys begin to walk and get more mobile, typically between the ages of 2 to 5. While they arent gene therapies, Sarepta also has two FDA-approved genetic medicines: Exondys51 (eteplirsen) and Vyondys53 (golodirsen). Now that the dystrophy gene was brought down to a useful size, the next challenge researchers faced was getting the gene therapy from the blood stream into the muscle. In mid-June, Sarepta announced that preliminary results from four boys ages 4-7 years were published in JAMA Neurology. This is based on a proprietary algorithm built from the drugs sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalDatas Pharmaceutical Intelligence Center. Duchenne Muscular Dystrophy (DMD) is an X-linked disease that is inherited. Founded in 2002, Alnylam has played a leading role in the translation of RNA interference (RNAi) into novel medicines. They are currently developing gene therapies for a range of diseases, including sickle cell disease and inherited blindness. With 125 participants enrolled, EMBARK is being proposed as the post-marketing confirmatory study for SRP-9001. The approved DMD therapies received a positive response and helped the patients. The BLA was supported by data from three studies: SRP-9001-101, SRP-9001-102 and SRP-9001-103. WebDuchenne Muscular Dystrophy (DMD) Core Dataset; Facioscapulohumeral Muscular Dystrophy (FSHD) Core Dataset DMD Research overview. It is currently being investigated in a Phase I/II study in six boys ages 4 and up. This loss adds up to about 50 billion yen, or about $390 million (U.S.). The Agency has also granted the companies priority review and set the regulatory action date for May 29, 2023. HuCo kidney, HuCo heart, HuCo islet cells, HuCo liver perfusion. Focuses on clinical-stage gene therapy. The trials main purpose is to monitor changes in NSAA scores. Sarepta Therapeutics obtains positive preliminary phase 1/2a results for patients with DMD using its gene therapy product. This type of gene therapy has the potential to pass the modified genes onto future generations, but it is still experimental and raises ethical concerns. Gene therapy is a medical treatment that involves modifying a person's genes or genetic material to treat or prevent disease. The mutated gene is on the X chromosome, making DMD an X-linked disease. With funding from biotech companies and the US Department of Defense, a blinded, placebo control study in dogs was approved. Moreover, the companies are hoping that their Duchenne Muscular Dystrophy treatment will slow or even stop disease progression, giving patients a chance to avoid the devastating effects of Duchenne. 1985 - 2023 BioSpace.com. According to the companys press release, preliminary data from nine boys with DMD (ages 6-12) showed the therapy was well-tolerated during intravenous infusion. Published: Nov 28, 2022 Duchenne Muscular Dystrophy is caused by a dystrophin gene mutation (a protein in the muscles). Afamitresgene autoleucel or afami-cel (formerly ADP-A2M4), ADP-A2M4CD8 SPEAR T-cell therapy. Duchenne Muscular Dystrophy has long been a promising candidate for gene therapy, but overcoming several difficult technical challenges has proven difficult. Sarepta Therapeutics said topline results from Part 2 of its study SRP-9001-102, an ongoing, randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of its gene therapy for the progressive neuromuscular condition Duchenne muscular dystrophy, showed statistically Duchenne muscular dystrophy effects all muscle cells, so an ideal therapy should target the whole body. We have developed other critical functions to ensure proper gene delivery. PF-06939926was granted Fast Track designation in 2020. Its experimental therapies are now in clinical trials for Gaucher disease type 1 and cystinosis. The company recently presented a clinical update at the virtual American Society of Gene and Cell Therapy (ASGCT) meeting in May. AAVs are also common viruses some people have already been exposed to AAVs naturally and would never know because they cause no symptoms. SRP-9001 aims to treat DMD by delivering a gene that codes for a functional copy of dystrophin to the muscle tissues. One of those labs, Louis Kunkels lab, identified the dystrophin gene first in 1986.. Can FcRn Antagonists Be The Game-Changer in the Generalized Myasthenia Gravis (gMG) Treatment Market? Get Sample Report: https://www.alliedmarketresearch.com/request-sample/2841. The company is pursuing various applications for metabolic, genetic diseases and oncology with its gene editing technologies. In 2019, it spent $4.3 billion to acquire gene therapy specialist Spark Therapeutics. Sareptas lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy, which is being evaluated in a phase I/II study for DMD. The leading companies developing gene therapy candidates for DMD are Sarepta Therapeutics, Roche, Pfizer, Solid Biosciences, and Regenxbio. Currently these trials are taking place in the US. MedTech 100 is a financial index calculated using the BIG100 companies covered in Autolus specializes in developing CAR-T cell therapies. It is administered by intravascular and intramuscular routes. Duchenne Muscular Dystrophy signs and symptoms include pelvic muscles atrophy, followed by involvement of the shoulder muscles. Stakeholders are hopeful a new gene therapy that utilizes magnetic resonance imaging (MRI) could be beneficial for adolescent patients with Duchenne Credit: Shutterstock, Engineering Natural Killer Cells for Cancer Immunotherapy [Video], Targeting the untargetable and treating the untreatable, Neural networks overcome the setbacks of current computational drug discovery, Copyright 1999-2023 John Wiley & Sons, Inc. All rights reserved. Get industry leading news, data and analysis delivered to your inbox. Which Countries Top the Chart in Global Pharmaceutical Market? His innovative Muscle-Targeted, Non-Viral platform has the potential to provide a novel gene therapy treatment for DMD a wide-range of other neuromuscular and cardiac disorders. Explore our blog to know more about Duchenne Muscular Dystrophy Treatment Market. The company has two approved chimeric antigen receptor (CAR T) cell therapies in hematologic malignancies that use two distinct targets targeting separate blood cancers. We discovered in 1997 that AAV vectors can be delivered to muscle cells and have a therapeutic benefit, said Byrne. AAV RP-A501, LV RP-L102, LV RP-L201, LV RP-L301. AAV is most often restricted to a single administration due to the likelihood of an immune response to the virus. Most boys stop walking and need a wheelchair between 9 and 14 years old.. Or higher doses to drive the virus into the muscles? Hesterlee added. Exploring Potential New Avenues for Bronchopulmonary Dysplasia Treatment, A Market Space Beyond Lucentis and Eylea for Retinal Vein Occlusion Treatment. Focuses on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. ISSN 2940-2034, systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy, Tiny, anti-inflammatory nanomotors to treat rheumatoid arthritis, Explaining the Universes accelerated expansion without dark energy, New material is a game changer in radiative cooling, A quark star may have just been discovered, Nanoparticles that self-assemble inside cells to fight cancer, James Webb Telescope images the Pillars of Creation, Anti-aging drug could help turn back the clock, Meteorite that struck a driveway in small UK town holds key ingredients for life. The Agency has also granted the therapy priority review and set the regulatory action date for May 29, 2023. It is using its STAR-D technology to develop topical treatments for rare or orphan dermatological indications. The biopharma is developing genome-edited off-the-shelf CAR-T and CAR-NK cell therapies for various tumor types. "The clinical evidence data for SRP-9001 represents the largest and broadest patient experience with a gene therapy for Duchenne," Tracy Sorrentino, executive director of corporate affairs, toldBioSpace. The patients body will react to the viral vector just like it would any other virus, creating antibodies to hunt and destroy the gene therapy viruses. LGMD2E -sarcoglycan, LGMD2D -sarcoglycan. The collaboration could be worth more than $3 billion. Next, the bad: interim data from the phase I/II Ignite DMD trial are disappointing, and the groups stock slid 24% this morning. July 6, 2022. The company is using a stem cell biology and genomics platform to develop a novel autologous induced pluripotent stem cell (iPSC)-derived neuron replacement therapy for Parkinsons Disease. The company also has a CRISPR alliance with Mammoth Biosciences to develop in vivo gene-editing therapies. Duchenne Muscular Dystrophy Treatment Outlook, Upcoming Potential Duchenne Muscular Dystrophy Gene Therapy, FAQ For Duchenne Muscular Dystrophy (DMD). Likelihood of Approval and Phase Transition Success Rate Mod IMT-009 by Immunitas Therapeutics for Squamous Non-Small Cell Lung Cancer: Likelihood of Approval, IMT-009 by Immunitas Therapeutics for Diffuse Large B-Cell Lymphoma: Likelihood of Approval, IMS-001 by Imstem Biotechnology for Relapsing Remitting Multiple Sclerosis (RRMS): Likelihood of Approval, IMS-001 by Imstem Biotechnology for Primary Progressive Multiple Sclerosis (PPMS): Likelihood of Approval. AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. The whole 2.2 Mb dystrophin gene over 440 times as big is too large to fit inside any AAV. FDA Approved: December 12, 2019; Company: Sarepta Therapeutics It has a pipeline of in vivo and ex vivo therapies. WebI am a licensed clinical therapist and provide customized therapy services for individuals, couples, and families." DMD is a progressive muscle wasting disease caused by a genetic mutation. This button displays the currently selected search type. In April, due to drug development challenges and fraught economic circumstances, the company wasforcedto slash its workforce by 35%. Sometimes called minidystrophins, there are slight variations between different versions of these shortened genes, but the key is they are all small enough to fit into AAV, explained Hesterlee. Gene over 440 times as big is too large to fit inside any aav is also developing for. Neurological disorders and other diseases Exondys51 ( eteplirsen ) and Vyondys53 ( golodirsen ) X-linked disease or afami-cel formerly! For rare or orphan dermatological indications Potential New Avenues for Bronchopulmonary Dysplasia Treatment, a,. 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